A newly identified disorder, MINA syndrome, results from a mutation in the NAMPT protein that deprives motor neurons of energy, leading to severe movement problems. The discovery not only deepens ...

The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...

A drug candidate found to protect nerve cells damaged by motor neuron disease (MND), could offer new hope to people living ...

The experimental therapy M102 slowed disease progression in preclinical experiments done in mouse and cell models of ALS, a ...

Novartis wins FDA approval for Itvisma, a one-time gene therapy offering broader SMA treatment with sustained motor function ...

The disease, called NAMPT axonopathy Mutation syndrome (MINA), causes damage to motor neurons, nerve cells that transmit signals from the brain and spinal cord to muscles. This is the result of a rare ...

By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers ...

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...

Researchers at Tel Aviv University led a large-scale international study that opens a new avenue for treating the fatal degenerative disease ALS, considered incurable until now. The researchers ...

Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...

Alice Wong, a nationally recognized disability rights advocate and author who founded the Disability Visibility Project, has ...

FDA cleared Itvisma as the first gene-replacement therapy available to people 2 and older with the rare muscle-weakening ...